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Article

Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

21 Aug 2019

Abstract excerpt

Niemann-Pick disease type A (NPD-A) is a lysosomal storage disorder characterized by neurodegeneration and early death. It is caused by loss-of-function mutations in the gene encoding for acid sphingomyelinase (ASM), which hydrolyzes sphingomyelin into ceramide. Here, we evaluated the safety of cerebellomedullary (CM) cistern injection of adeno-associated viral vector serotype 9 encoding human ASM (AAV9-hASM) in...

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PMID: 31434754
DOI: 10.1126/scitranslmed.aat3738

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